ICER Publishes Final Evidence Report on Gene Therapies for Sickle Cell Disease
— Despite uncertainty regarding longer-term outcomes, lovo-cel and exa-cel both estimated to produce substantial gains in length and qualit…
— Despite uncertainty regarding longer-term outcomes, lovo-cel and exa-cel both estimated to produce substantial gains in length and qualit…
AMP ALS, the latest public-private partnership in the FNIH’s growing AMP portfolio, will centralize and build the largest data source for ALS research. This …
The Accelerating Medicines Partnership® (AMP®) Amyotrophic Lateral Sclerosis (AMP ALS) is accelerating therapeutic and biomarker development for familial and sporadic ALS by addressing the challenges…
Nature Reviews Neurology – In this Perspective, the authors propose a new classification system for amyotrophic lateral sclerosis and related neurodegenerative disorders that recognizes, in…
Plan ahead. Actively listen. And discuss how to move forward.
Nora Kenworthy has spent years studying the world of crowdfunding for health care — along with the way it ends up amplifying the inequities that…
The Elon Musk-backed startup has proposed solutions for a thread pullout problem experienced by its first participant, Noland Arbaugh.
Guidance for developing drugs to treat early Alzheimer@s disease should focus on factors such as cognitive changes and brain atrophy, according to comments submitted to…
A local man is thankful for the groundbreaking research happening in a lab at the Stowers Institute in Kansas City, Missouri.
NIH-led findings point to a biomarker for rare disorders associated with the gene PNPLA6.
A new medication is offering some ALS patients more hope than ever. It’s actually slowing the disease down in some people like Jessica Morris.